
This is Mael, a four and a half year old Swiss boy, affected by the ultra rare and fatal storage disease, Niemann Pick Type C (NPC). Mael is a very happy and wonderful boy and does not know about his uncertain and sad future. NPC will slowly [...]
Building awareness and communities to support and find treatments for Rare Diseases and Genetic Conditions

This is Mael, a four and a half year old Swiss boy, affected by the ultra rare and fatal storage disease, Niemann Pick Type C (NPC). Mael is a very happy and wonderful boy and does not know about his uncertain and sad future. NPC will slowly [...]

The LSD Support Society of India is a group comprised of patients, families, and caregivers who know what it’s like to live with Lysosomal Storage Disorders (LSDs). If you have never heard of lysosomal storage diseases, they are group of approximately 40 rare inherited metabolic [...]

Case was only 4 hours old when he first went on a ventilator. His young life was peppered by minor symptoms that sent him to one specialist after another. A swallow study, a head ultrasound, adenoids removed, pectus excavatum, laryngomalacia, uncontrollable diarrhea, and falling. It [...]

Our 4 year old son Joseph Colton has Niemann-Pick disease type A/B. The Niemann-Pick (NP) diseases belong to a family of 40+ inherited disorders identified as lysosomal storage diseases or LSDs. There are two distinct sub-families of Niemann Pick diseases. NPA and NPB diseases are [...]

Our 4 year old son Joseph Colton has Niemann-Pick disease type A/B. The Niemann-Pick (NP) diseases belong to a family of 40+ inherited disorders identified as lysosomal storage diseases or LSDs. There are two distinct sub-families of Niemann Pick diseases. NPA and NPB diseases are [...]

I received a call today at The Hide and Seek Foundation from a woman who’s cousin’s seven month old son, Ben, has been displaying possible symptoms of a neurometabolic disease. You know, darting eyes, not rolling over, trunk weakness. He went to a neurologist yesterday [...]
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